JP2018532721A5 - - Google Patents

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JP2018532721A5
JP2018532721A5 JP2018514267A JP2018514267A JP2018532721A5 JP 2018532721 A5 JP2018532721 A5 JP 2018532721A5 JP 2018514267 A JP2018514267 A JP 2018514267A JP 2018514267 A JP2018514267 A JP 2018514267A JP 2018532721 A5 JP2018532721 A5 JP 2018532721A5
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nanoparticle composition
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glycero
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JP6948313B2 (en
JP6948313B6 (en
JP2018532721A (en
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Priority claimed from PCT/US2016/052352 external-priority patent/WO2017049245A2/en
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Priority to JP2021151758A priority Critical patent/JP7326395B2/en
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Publication of JP6948313B6 publication Critical patent/JP6948313B6/en
Priority to JP2023125995A priority patent/JP2023164798A/en
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式（ＩＡ）：
（式中、
ｌが、１、２、３、４、および５から選択され；
ｍが、５、６、７、８、および９から選択され；
Ｍ１が、結合またはＭ’であり；
Ｒ４が、非置換Ｃ１〜３アルキル、または−（ＣＨ２）ｎＱであり、ここで、Ｑが、ＯＨ、−ＮＨＣ（Ｓ）Ｎ（Ｒ）２、−ＮＨＣ（Ｏ）Ｎ（Ｒ）２、−Ｎ（Ｒ）Ｃ（Ｏ）Ｒ、−Ｎ（Ｒ）Ｓ（Ｏ）２Ｒ、−Ｎ（Ｒ）Ｒ８、−ＮＨＣ（＝ＮＲ９）Ｎ（Ｒ）２、−ＮＨＣ（＝ＣＨＲ９）Ｎ（Ｒ）２、−ＯＣ（Ｏ）Ｎ（Ｒ）２、−Ｎ（Ｒ）Ｃ（Ｏ）ＯＲ、−Ｎ（ＯＲ）Ｃ（Ｏ）Ｒ、−Ｎ（ＯＲ）Ｓ（Ｏ）２Ｒ、−Ｎ（ＯＲ）Ｃ（Ｏ）ＯＲ、−Ｎ（ＯＲ）Ｃ（Ｏ）Ｎ（Ｒ）２、−Ｎ（ＯＲ）Ｃ（Ｓ）Ｎ（Ｒ）２、−Ｎ（ＯＲ）Ｃ（＝ＮＲ９）Ｎ（Ｒ）２、−Ｎ（ＯＲ）Ｃ（＝ＣＨＲ９）Ｎ（Ｒ）２、またはヘテロアリールであり、および、各ｎが、１、２、３、４、または５から選択され；
ＭおよびＭ’が、独立して、−Ｃ（Ｏ）Ｏ−、−ＯＣ（Ｏ）−、−Ｃ（Ｏ）Ｎ（Ｒ’）−、−Ｐ（Ｏ）（ＯＲ’）Ｏ−、−Ｓ−Ｓ−、アリール基、およびヘテロアリール基から選択され；
Ｒ２およびＲ３が両方とも、Ｃ１〜１４アルキル、またはＣ２〜１４アルケニルであり；
Ｒ ８ が、Ｃ ３〜６ 炭素環および複素環からなる群から選択され；
Ｒ ９ が、Ｈ、ＣＮ、ＮＯ ２ 、Ｃ １〜６ アルキル、−ＯＲ、−Ｓ（Ｏ） ２ Ｒ、−Ｓ（Ｏ） ２ Ｎ（Ｒ） ２ 、Ｃ ２〜６ アルケニル、Ｃ ３〜６ 炭素環および複素環からなる群から選択され；
各Ｒが、独立して、Ｃ １〜３ アルキル、Ｃ ２〜３ アルケニル、およびＨからなる群から選択され；
Ｒ’が、直鎖状アルキルである）
の化合物またはその塩もしくは異性体。 Formula (IA):
(Where
l is selected from 1, 2, 3, 4, and 5;
m is selected from 5, 6, 7, 8, and 9;
M 1 is a bond or M ′;
R 4 is unsubstituted C 1-3 alkyl, or — (CH 2 ) n Q, where Q is OH, —NHC (S) N (R) 2 , —NHC (O) N (R ) 2 , —N (R) C (O) R, —N (R) S (O) 2 R, —N (R) R 8 , —NHC (═NR 9 ) N (R) 2 , —NHC ( = CHR 9) N (R) 2, -OC (O) N (R) 2, -N (R) C (O) OR, -N (OR) C (O) R, -N (OR) S ( O) 2 R, -N (OR ) C (O) OR, -N (OR) C (O) N (R) 2, -N (OR) C (S) N (R) 2, -N (OR ) C (= NR 9) n (R) 2, -N (oR) C (= CHR 9) n (R) 2, or a heteroarylene Le, and each n is 1, 2, 3, 4, Or selected from 5 ;
M and M ′ are independently —C (O) O—, —OC (O) —, —C (O) N (R ′) —, —P (O) (OR ′) O—, — Selected from S—S—, aryl groups, and heteroaryl groups;
R 2 and R 3 are both C 1-14 alkyl, or C 2-14 alkenyl ;
R 8 is selected from the group consisting of C 3-6 carbocycles and heterocycles;
R 9 is H, CN, NO 2 , C 1-6 alkyl, —OR, —S (O) 2 R, —S (O) 2 N (R) 2 , C 2-6 alkenyl, C 3-6. Selected from the group consisting of carbocycle and heterocycle;
Each R is independently selected from the group consisting of C 1-3 alkyl, C 2-3 alkenyl, and H;
R ′ is linear alkyl )
Or a salt or isomer thereof . Ｒ  R ４4 が、非置換メチルまたは−（ＣＨIs unsubstituted methyl or-(CH ２2 ）) ｎn Ｑであり、ここで、Ｑが、ＯＨ、−ＮＨＣ（Ｓ）Ｎ（ＣＨQ, where Q is OH, —NHC (S) N (CH ３3 ）) ２2 、−ＮＨＣ（Ｓ）ＮＨ（ＣＨ, -NHC (S) NH (CH ３3 ）、−ＮＨＣ（Ｏ）ＮＨ（ＣＨ), -NHC (O) NH (CH ３3 ）、または−ＮＨＣ（Ｏ）Ｎ（ＣＨ), Or —NHC (O) N (CH ３3 ）) ２2 である、請求項１に記載の化合物。The compound of claim 1, wherein 式（Ｉ）：  Formula (I):
（式中、(Where
Ｒ  R １1 が、ＣBut C ５〜３０5-30 アルキル、ＣAlkyl, C ５〜２０5-20 アルケニル、−ＲAlkenyl, -R ＊* ＹＲ”、−ＹＲ”、および−Ｒ”Ｍ’Ｒ’からなる群から選択され；Selected from the group consisting of YR ", -YR", and -R "M'R ';
Ｒ  R ２2 およびＲAnd R ３3 が、独立して、Ｈ、ＣBut independently H, C １〜１４1-14 アルキル、ＣAlkyl, C ２〜１４2-14 アルケニル、−ＲAlkenyl, -R ＊* ＹＲ”、−ＹＲ”、および−ＲYR ", -YR", and -R ＊* ＯＲ”からなる群から選択され、またはＲOR "or R ２2 およびＲAnd R ３3 が、それらが結合される原子と一緒に、複素環または炭素環を形成し；Form together with the atoms to which they are attached a heterocycle or carbocycle;
各Ｒ  Each R ５5 が、独立して、ＣBut independently, C １〜３1-3 アルキル、ＣAlkyl, C ２〜３2-3 アルケニル、およびＨからなる群から選択され；Selected from the group consisting of alkenyl and H;
各Ｒ  Each R ６6 が、独立して、ＣBut independently, C １〜３1-3 アルキル、ＣAlkyl, C ２〜３2-3 アルケニル、およびＨからなる群から選択され；Selected from the group consisting of alkenyl and H;
ＭおよびＭ’が、独立して、−Ｃ（Ｏ）Ｏ−、−ＯＣ（Ｏ）−、−ＯＣ（Ｏ）−Ｍ”−Ｃ（Ｏ）Ｏ−、−Ｃ（Ｏ）Ｎ（Ｒ’）−、−Ｎ（Ｒ’）Ｃ（Ｏ）−、−Ｃ（Ｏ）−、−Ｃ（Ｓ）−、−Ｃ（Ｓ）Ｓ−、−ＳＣ（Ｓ）−、−ＣＨ（ＯＨ）−、−Ｐ（Ｏ）（ＯＲ’）Ｏ−、−Ｓ（Ｏ）  M and M ′ are independently —C (O) O—, —OC (O) —, —OC (O) —M ″ —C (O) O—, —C (O) N (R ′ )-, -N (R ') C (O)-, -C (O)-, -C (S)-, -C (S) S-, -SC (S)-, -CH (OH)- , -P (O) (OR ') O-, -S (O) ２2 −、−Ｓ−Ｓ−、アリール基、およびヘテロアリール基から選択され、ここでＭ”が、結合、Ｃ-, -S-S-, an aryl group, and a heteroaryl group, wherein M "is a bond, C １〜１３1-13 アルキル、またはＣAlkyl or C ２〜１３2-13 アルケニルであり；Is alkenyl;
Ｒ  R ７7 が、ＣBut C １〜３1-3 アルキル、ＣAlkyl, C ２〜３2-3 アルケニル、およびＨからなる群から選択され；Selected from the group consisting of alkenyl and H;
各Ｒが、独立して、Ｈ、Ｃ  Each R is independently H, C １〜３1-3 アルキル、およびＣAlkyl and C ２〜３2-3 アルケニルからなる群から選択され；Selected from the group consisting of alkenyl;
各Ｒ’が、独立して、Ｃ  Each R 'is independently C １〜１８1-18 アルキル、ＣAlkyl, C ２〜１８2-18 アルケニル、−ＲAlkenyl, -R ＊* ＹＲ”、−ＹＲ”、およびＨからなる群から選択され；Selected from the group consisting of YR ″, —YR ″, and H;
各Ｒ”が、独立して、Ｃ  Each R ″ is independently C ３〜１５3-15 アルキルおよびＣAlkyl and C ３〜１５3-15 アルケニルからなる群から選択され；Selected from the group consisting of alkenyl;
各Ｒ  Each R ＊* が、独立して、ＣBut independently, C １〜１２1-12 アルキルおよびＣAlkyl and C ２〜１２2-12 アルケニルからなる群から選択され；Selected from the group consisting of alkenyl;
各Ｙが、独立して、Ｃ  Each Y is independently C ３〜６3-6 炭素環であり；A carbocycle;
各Ｘが、独立して、Ｆ、Ｃｌ、Ｂｒ、およびＩからなる群から選択され；  Each X is independently selected from the group consisting of F, Cl, Br, and I;
Ｒ  R １０10 が、アルキルアミノおよびジアルキルアミノからなる群から選択され；Is selected from the group consisting of alkylamino and dialkylamino;
ｍが、５、６、７、８、９、１０、１１、１２、および１３から選択され；  m is selected from 5, 6, 7, 8, 9, 10, 11, 12, and 13;
ｎが、１、２、３、４、および５から選択される）  n is selected from 1, 2, 3, 4, and 5)
の化合物またはそのＮオキシドもしくは塩。Or an N oxide or salt thereof. ｎが、２または３である、請求項３に記載の化合物。  4. A compound according to claim 3, wherein n is 2 or 3. Ｒ  R １０10 が、アルキルアミノである、請求項３または４に記載の化合物。5. The compound according to claim 3 or 4, wherein is alkylamino. Ｒ  R １０10 が、メチルアミノおよびジメチルアミノからなる群から選択される、請求項５に記載の化合物。6. The compound of claim 5, wherein is selected from the group consisting of methylamino and dimethylamino. 式（ＶＩＩＩａ）：  Formula (VIIIa):
（式中、(Where
ｌが、１、２、３、４、および５から選択され；  l is selected from 1, 2, 3, 4, and 5;
ｏが、３、４、５、６、７、８、９、１０、および１１から選択され；  o is selected from 3, 4, 5, 6, 7, 8, 9, 10, and 11;
Ｍ  M １1 が、Ｍ’であり；Is M ';
ＭおよびＭ’が、独立して、−Ｃ（Ｏ）Ｏ−および−ＯＣ（Ｏ）−から選択され；  M and M 'are independently selected from -C (O) O- and -OC (O)-;
Ｒ  R １０10 が、アルキルアミノおよびジアルキルアミノからなる群から選択され；Is selected from the group consisting of alkylamino and dialkylamino;
Ｒ  R ２2 およびＲAnd R ３3 が、両方とも、ＣBut both are C １〜１４1-14 アルキル、またはＣAlkyl or C ２〜１４2-14 アルケニルであり；Is alkenyl;
Ｒ’が、Ｃ  R 'is C １〜１８1-18 アルキルである）Is alkyl)
の化合物またはその塩。Or a salt thereof. ｏが、５である、請求項７に記載の化合物。  8. A compound according to claim 7, wherein o is 5. Ｍ’が、−Ｃ（Ｏ）Ｏである、請求項１〜８のいずれか一項に記載の化合物。  The compound according to any one of claims 1 to 8, wherein M 'is -C (O) O. 前記化合物が、式（ＩＩ）：
（式中、
ｌが、１、２、３、４、および５から選択される）
の化合物またはその塩もしくは異性体である、請求項１に記載の化合物。 Said compound is of formula (II):
(Where
l is selected from 1, 2, 3, 4, and 5)
The compound or its salts or isomers, the compounds of claim 1. Ｒ  R ４4 が、−（ＣＨIs-(CH ２2 ）) ｎn Ｑであり、ここで、Ｑが、ＯＨであり、ｎが、１、２、３、４、または５から選択され；Q, wherein Q is OH and n is selected from 1, 2, 3, 4, or 5;
ＭおよびＭ  M and M １1 が、独立して、−Ｃ（Ｏ）Ｏ−、および−ＯＣ（Ｏ）−から選択され；Are independently selected from -C (O) O- and -OC (O)-;
Ｒ  R ２2 およびＲAnd R ３3 が、両方とも、ＣBut both are C １〜１４1-14 アルキル、またはＣAlkyl or C ２〜１４2-14 アルケニルであり；Is alkenyl;
Ｒ’が、Ｃ  R 'is C １〜１２1-12 直鎖状アルキルである、請求項１０に記載の化合物。11. A compound according to claim 10, which is a linear alkyl. ｌが、３、４、および５から選択される、請求項１，２，および７〜１１のいずれか一項に記載の化合物。  12. A compound according to any one of claims 1, 2, and 7-11, wherein l is selected from 3, 4, and 5. Ｍが、−Ｃ（Ｏ）Ｏ−である、請求項１〜１２のいずれか一項に記載の化合物。  The compound according to any one of claims 1 to 12, wherein M is -C (O) O-. 前記化合物が、式（ＩＩｄ）  Said compound is of formula (IId)
の化合物、またはその塩もしくは異性体であり、Or a salt or isomer thereof,
ここで、ｎが、２、３、および４から選択される、請求項１に記載の化合物。2. The compound according to claim 1, wherein n is selected from 2, 3, and 4. ｎが、２または３である、請求項１４に記載の化合物。  15. A compound according to claim 14, wherein n is 2 or 3. Ｒ  R ２2 およびＲAnd R ３3 が、同じである、請求項１〜１５のいずれか一項に記載の化合物。16. The compound according to any one of claims 1 to 15, wherein are the same. Ｒ  R ２2 およびＲAnd R ３3 が、ＣBut C ８8 アルキルである、請求項１６に記載の化合物。17. A compound according to claim 16 which is alkyl. Ｒ  R ２2 およびＲAnd R ３3 が、異なる、請求項１〜１５のいずれか一項に記載の化合物。16. The compound according to any one of claims 1 to 15, wherein are different. Ｒ’が、Ｃ  R 'is C ９〜１１9-11 アルキルである、請求項１〜１８のいずれか一項に記載の化合物。19. A compound according to any one of claims 1 to 18 which is alkyl. Ｒ’が、Ｃ  R 'is C ９9 アルキル、またはＣAlkyl or C １１11 アルキルである、請求項１９に記載の化合物。20. A compound according to claim 19 which is alkyl. 前記化合物が、式（ＩＩａ）
の化合物、またはその塩もしくは異性体である、請求項１または２に記載の化合物。 Said compound is of formula (IIa)
The compound of Claim 1 or 2 which is a compound of this, or its salt or isomer. 前記化合物が、（式Ａ）、（式Ｂ）、（式Ｃ）、または（式Ｄ）：  The compound is (Formula A), (Formula B), (Formula C), or (Formula D):
の化合物、またはその塩である、請求項１に記載の化合物。The compound of Claim 1 which is a compound of these, or its salt. 前記化合物が、（式Ｅ）または（式Ｆ）：  The compound is represented by (formula E) or (formula F):
の化合物、またはその塩である、請求項３に記載の化合物。The compound of Claim 3 which is a compound of these, or its salt. 前記化合物が、化合物１〜５、７、８、１９〜２０、２３、２８、３０、３２〜４７、５６〜６１、９６、１００、１０４、１０７〜１１７、１２２〜１２４、１２６〜１２９、１３１、１３３〜１３４、１４３〜１４７、１５３、１６８〜１７０、１７３〜１７９、１８１〜１８２、１８５、１８６、１９３、１９４、１９６〜１９８、２００、２０３〜２１３、２１５、２１８〜２２８、２３０、および２３１からなる群から選択される化合物、またはその塩もしくは異性体である、請求項１に記載の化合物。  The compound is compound 1-5, 7, 8, 19-20, 23, 28, 30, 32-47, 56-61, 96, 100, 104, 107-117, 122-124, 126-129, 131. 133-134, 143-147, 153, 168-170, 173-179, 181-182, 185, 186, 193, 194, 196-198, 200, 203-213, 215, 218-228, 230, and The compound according to claim 1, which is a compound selected from the group consisting of 231 or a salt or isomer thereof. 前記塩が、薬学的に許容できる塩である、請求項１〜２４のいずれか一項に記載の化合物。  25. A compound according to any one of claims 1 to 24, wherein the salt is a pharmaceutically acceptable salt. 請求項１〜２５のいずれか一項に記載の化合物を含む脂質成分を含むナノ粒子組成物。 A nanoparticle composition comprising a lipid component comprising the compound according to any one of claims 1 to 25 . 前記脂質成分が、リン脂質をさらに含む、請求項２６に記載のナノ粒子組成物。 27. The nanoparticle composition of claim 26 , wherein the lipid component further comprises a phospholipid. 前記リン脂質が、１，２−ジリノレオイル−ｓｎ−グリセロ−３−ホスホコリン（ＤＬＰＣ）、１，２−ジミリストイル−ｓｎ−グリセロ−ホスホコリン（ＤＭＰＣ）、１，２−ジオレオイル−ｓｎ−グリセロ−３−ホスホコリン（ＤＯＰＣ）、１，２−ジパルミトイル−ｓｎ−グリセロ−３−ホスホコリン（ＤＰＰＣ）、１，２−ジステアロイル−ｓｎ−グリセロ−３−ホスホコリン（ＤＳＰＣ）、１，２−ジウンデカノイル−ｓｎ−グリセロ−ホスホコリン（ＤＵＰＣ）、１−パルミトイル−２−オレオイル−ｓｎ−グリセロ−３−ホスホコリン（ＰＯＰＣ）、１，２−ジ−Ｏ−オクタデセニル−ｓｎ−グリセロ−３−ホスホコリン（１８：０ジエーテルＰＣ）、１−オレオイル−２−コレステリルヘミスクシノイル−ｓｎ−グリセロ−３−ホスホコリン（ＯＣｈｅｍｓＰＣ）、１−ヘキサデシル−ｓｎ−グリセロ−３−ホスホコリン（Ｃ１６ Ｌｙｓｏ ＰＣ）、１，２−ジリノレノイル−ｓｎ−グリセロ−３−ホスホコリン、１，２−ジアラキドノイル−ｓｎ−グリセロ−３−ホスホコリン、１，２−ジドコサヘキサエノイル−ｓｎ−グリセロ−３−ホスホコリン，１，２−ジオレオイル−ｓｎ−グリセロ−３−ホスホエタノールアミン（ＤＯＰＥ）、１，２−ジフィタノイル−ｓｎ−グリセロ−３−ホスホエタノールアミン（ＭＥ １６．０ ＰＥ）、１，２−ジステアロイル−ｓｎ−グリセロ−３−ホスホエタノールアミン、１，２−ジリノレオイル−ｓｎ−グリセロ−３−ホスホエタノールアミン、１，２−ジリノレノイル−ｓｎ−グリセロ−３−ホスホエタノールアミン、１，２−ジアラキドノイル−ｓｎ−グリセロ−３−ホスホエタノールアミン、１，２−ジドコサヘキサエノイル−ｓｎ−グリセロ−３−ホスホエタノールアミン、１，２−ジオレオイル−ｓｎ−グリセロ−３−ホスホ−ｒａｃ−（１−グリセロール）ナトリウム塩（ＤＯＰＧ）、スフィンゴミエリン、およびそれらの混合物からなる群から選択される、請求項２７に記載のナノ粒子組成物。 The phospholipid is 1,2-dilinoleoyl-sn-glycero-3-phosphocholine (DLPC), 1,2-dimyristoyl-sn-glycero-phosphocholine (DMPC), 1,2-dioleoyl-sn-glycero-3- Phosphocholine (DOPC), 1,2-dipalmitoyl-sn-glycero-3-phosphocholine (DPPC), 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC), 1,2-diundecanoyl-sn-glycero -Phosphocholine (DUPC), 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine (POPC), 1,2-di-O-octadecenyl-sn-glycero-3-phosphocholine (18: 0 diether PC) 1-oleoyl-2-cholesteryl hemisuccinoyl-sn-glycero- -Phosphocholine (OCchemsPC), 1-hexadecyl-sn-glycero-3-phosphocholine (C16 Lyso PC), 1,2-dilinolenoyl-sn-glycero-3-phosphocholine, 1,2-diachidonoyl-sn-glycero-3-phosphocholine 1,2-didocosahexaenoyl-sn-glycero-3-phosphocholine, 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine (DOPE), 1,2-diphytanoyl-sn-glycero-3- Phosphoethanolamine (ME 16.0 PE), 1,2-distearoyl-sn-glycero-3-phosphoethanolamine, 1,2-dilinoleoyl-sn-glycero-3-phosphoethanolamine, 1,2-dilinolenoyl- sn-glycero-3-phosphoethanolamine 1,2-diarachidonoyl-sn-glycero-3-phosphoethanolamine, 1,2-didocosahexaenoyl-sn-glycero-3-phosphoethanolamine, 1,2-dioleoyl-sn-glycero-3-phospho- 28. The nanoparticle composition of claim 27 , selected from the group consisting of rac- (1-glycerol) sodium salt (DOPG), sphingomyelin, and mixtures thereof. 前記リン脂質がＤＳＰＣまたはＤＯＰＥである、請求項２７に記載のナノ粒子組成物。 28. The nanoparticle composition of claim 27 , wherein the phospholipid is DSPC or DOPE. 前記脂質成分が、構造脂質をさらに含む、請求項２６〜２９のいずれか一項に記載のナノ粒子組成物。 30. The nanoparticle composition according to any one of claims 26 to 29 , wherein the lipid component further comprises a structured lipid. 前記構造脂質が、コレステロール、フェコステロール、シトステロール、エルゴステロール、カンペステロール、スチグマステロール、ブラシカステロール、トマチジン、ウルソール酸、α−トコフェロール、およびそれらの混合物からなる群から選択される、請求項３０に記載のナノ粒子組成物。 Said structural lipid, cholesterol, Fe Coste roll, sitosterol, ergosterol, campesterol, stigmasterol, brassicasterol, tomatidine, ursolic acid, alpha-tocopherol, and are selected from the group consisting of a mixture thereof, according to claim 30 The nanoparticle composition according to 1. 前記構造脂質がコレステロールである、請求項３１に記載のナノ粒子組成物。 32. The nanoparticle composition of claim 31 , wherein the structured lipid is cholesterol. 前記脂質成分が、ＰＥＧ脂質をさらに含む、請求項２６〜３２のいずれか一項に記載のナノ粒子組成物。 33. The nanoparticle composition according to any one of claims 26 to 32 , wherein the lipid component further comprises PEG lipids. 前記ＰＥＧ脂質が、ＰＥＧ−修飾ホスファチジルエタノールアミン、ＰＥＧ−修飾ホスファチジン酸、ＰＥＧ−修飾セラミド、ＰＥＧ−修飾ジアルキルアミン、ＰＥＧ−修飾ジアシルグリセロール、ＰＥＧ−修飾ジアルキルグリセロール、およびそれらの混合物からなる群から選択される、請求項３３に記載のナノ粒子組成物。 The PEG lipid is selected from the group consisting of PEG-modified phosphatidylethanolamine, PEG-modified phosphatidic acid, PEG-modified ceramide, PEG-modified dialkylamine, PEG-modified diacylglycerol, PEG-modified dialkylglycerol, and mixtures thereof. 34. The nanoparticle composition of claim 33 . 前記脂質成分が、カチオン性および／またはイオン性脂質をさらに含む、請求項２６〜３４のいずれか一項に記載のナノ粒子組成物。  35. A nanoparticle composition according to any one of claims 26 to 34, wherein the lipid component further comprises cationic and / or ionic lipids. 前記カチオン性および／またはイオン性脂質が、３−（ジドデシルアミノ）−Ｎ１，Ｎ１，４−トリドデシル−１−ピペラジンエタンアミン（ＫＬ１０）、Ｎ１−［２−（ジドデシルアミノ）エチル］−Ｎ１，Ｎ４，Ｎ４−トリドデシル−１，４−ピペラジンジエタンアミン（ＫＬ２２）、１４，２５−ジトリデシル−１５，１８，２１，２４−テトラアザ−オクタトリアコンタン（ＫＬ２５）、１，２−ジリノレイルオキシ−Ｎ，Ｎ−ジメチルアミンプロパン（ＤＬｉｎ−ＤＭＡ）、２，２−ジリノレイル−４−ジメチルアミノメチル−［１，３］−ジオキソラン（ＤＬｉｎ−Ｋ−ＤＭＡ）、ヘプタトリアコンタ−６，９，２８，３１−テトラエン−１９−イル４−（ジメチルアミノ）ブタノエート（ＤＬｉｎ−ＭＣ３−ＤＭＡ）、２，２−ジリノレイル−４−（２−ジメチルアミノエチル）−［１，３］−ジオキソラン（ＤＬｉｎ−ＫＣ２−ＤＭＡ）、１，２−ジオレイルオキシ−Ｎ，Ｎ−ジメチルアミンプロパン（ＤＯＤＭＡ）、２−（｛８−［（３β）−コレスタ−５−エン−３−イルオキシ］オクチル｝オキシ）−Ｎ，Ｎ−ジメチル−３−［（９Ｚ，１２Ｚ）−オクタデカ−９，１２−ジエン−１−イルオキシ］プロパン−１−アミン（オクチル−ＣＬｉｎＤＭＡ）、（２Ｒ）−２−（｛８−［（３β）−コレスタ−５−エン−３−イルオキシ］オクチル｝オキシ）−Ｎ，Ｎ−ジメチル−３−［（９Ｚ，１２Ｚ）−オクタデカ−９，１２−ジエン−１−イルオキシ］プロパン−１−アミン（オクチル−ＣＬｉｎＤＭＡ（２Ｒ））、および（２Ｓ）−２−（｛８−［（３β）−コレスタ−５−エン−３−イルオキシ］オクチル｝オキシ）−Ｎ，Ｎ−ジメチル−３−［（９Ｚ，１２Ｚ）−オクタデカ−９，１２−ジエン−１−イルオキシ］プロパン−１−アミン（オクチル−ＣＬｉｎＤＭＡ（２Ｓ））からなる群から選択される、請求項３５に記載のナノ粒子組成物。 The cationic and / or ionic lipid is 3- (didodecylamino) -N1, N1,4-tridodecyl-1-piperazineethanamine (KL10), N1- [2- (didodecylamino) ethyl] -N1. , N4, N4-tridodecyl-1,4-piperazinediethanamine (KL22), 14,25-ditridecyl-15,18,21,24-tetraaza-octatriacontane (KL25), 1,2-dilinoleyloxy -N, N-dimethylaminepropane (DLin-DMA), 2,2-dilinoleyl-4-dimethylaminomethyl- [1,3] -dioxolane (DLin-K-DMA), heptatriaconta-6,9,28 , 31-Tetraen-19-yl 4- (dimethylamino) butanoate (DLin-MC3-DMA), 2,2 Dilinoleyl-4- (2-dimethylaminoethyl)-[1,3] -dioxolane (DLin-KC2-DMA), 1,2-dioleyloxy-N, N-dimethylaminepropane (DODMA), 2-({ 8-[(3β) -Cholesta-5-en-3-yloxy] octyl} oxy) -N, N-dimethyl-3-[(9Z, 12Z) -octadeca-9,12-dien-1-yloxy] propane -1-amine (octyl-CLinDMA), (2R) -2-({8-[(3β) -cholest-5-en-3-yloxy] octyl} oxy) -N, N-dimethyl-3-[( 9Z, 12Z) -octadeca-9,12-dien-1-yloxy] propan-1-amine (octyl-CLinDMA (2R)), and (2S) -2-({8-[(3β) Cholesta-5-en-3-yloxy] octyl} oxy) -N, N-dimethyl-3-[(9Z, 12Z) -octadec-9,12-dien-1-yloxy] propan-1-amine (octyl- Ru is selected from the group consisting of CLinDMA (2S)), the nanoparticle composition of claim 35. 前記脂質成分が、３０ｍｏｌ％〜６０ｍｏｌ％の前記化合物、０ｍｏｌ％〜３０ｍｏｌ％のリン脂質、１８．５ｍｏｌ％〜４８．５ｍｏｌ％の構造脂質、および０ｍｏｌ％〜１０ｍｏｌ％のＰＥＧ脂質を含む、請求項２６〜３６のいずれか一項に記載のナノ粒子組成物。 Wherein the lipid component, the compound of 30mol% ~6 0mol%, 0 mol % ~3 0mol% phospholipids, 1 structured lipids of 8.5 mol% to 4 8.5 mol%, and 0 mol% to 1 0 mol 37. The nanoparticle composition according to any one of claims 26 to 36 , comprising% PEG lipid. 前記脂質成分が、５０ｍｏｌ％の前記化合物、１０ｍｏｌ％のリン脂質、３８．５ｍｏｌ％の構造脂質、および１．５ｍｏｌ％のＰＥＧ脂質を含む、請求項３７に記載のナノ粒子組成物。 Wherein the lipid component, said compound 5 0 mol%, 1 0 mol% of phospholipids, 3 8.5 mol% of the structural lipid, and 1. 38. The nanoparticle composition of claim 37 , comprising 5 mol% PEG lipid. 治療薬および／または予防薬をさらに含む、請求項２６〜３８のいずれか一項に記載のナノ粒子組成物。 The nanoparticle composition according to any one of claims 26 to 38 , further comprising a therapeutic agent and / or a prophylactic agent. 前記治療薬および／または予防薬が核酸である、請求項３９に記載のナノ粒子組成物。 40. The nanoparticle composition of claim 39 , wherein the therapeutic and / or prophylactic agent is a nucleic acid. 前記治療薬および／または予防薬がリボ核酸（ＲＮＡ）である、請求項４０に記載のナノ粒子組成物。 41. The nanoparticle composition of claim 40 , wherein the therapeutic and / or prophylactic agent is ribonucleic acid (RNA). 前記ＲＮＡが、低分子干渉ＲＮＡ（ｓｉＲＮＡ）、非対称干渉ＲＮＡ（ａｉＲＮＡ）、マイクロＲＮＡ（ｍｉＲＮＡ）、ダイサー基質ＲＮＡ（ｄｓＲＮＡ）、小ヘアピンＲＮＡ（ｓｈＲＮＡ）、メッセンジャーＲＮＡ（ｍＲＮＡ）、およびそれらの混合物からなる群から選択される、請求項４１に記載のナノ粒子組成物。 The RNA is from small interfering RNA (siRNA), asymmetric interfering RNA (aiRNA), micro RNA (miRNA), dicer substrate RNA (dsRNA), small hairpin RNA (shRNA), messenger RNA (mRNA), and mixtures thereof. 42. The nanoparticle composition of claim 41 , selected from the group consisting of: 前記ＲＮＡがｍＲＮＡである、請求項４１または４２に記載のナノ粒子組成物。 43. The nanoparticle composition of claim 41 or 42 , wherein the RNA is mRNA. 前記ｍＲＮＡが、ステムループ、鎖終止ヌクレオシド、ポリＡ配列、ポリアデニル化シグナル、および／または５’キャップ構造の１つ以上を含む、請求項４３に記載のナノ粒子組成物。 44. The nanoparticle composition of claim 43 , wherein the mRNA comprises one or more of a stem loop, a chain termination nucleoside, a poly A sequence, a polyadenylation signal, and / or a 5 'cap structure. 前記治療薬および／または予防薬の封入効率が、少なくとも８０％または少なくとも９０％である、請求項３９〜４４のいずれか一項に記載のナノ粒子組成物。 45. The nanoparticle composition according to any one of claims 39 to 44 , wherein the encapsulation efficiency of the therapeutic and / or prophylactic agent is at least 80% or at least 90% . 前記治療薬および／または予防薬に対する前記脂質成分のｗｔ／ｗｔ比が、１０：１〜６０：１である、請求項３９〜４５のいずれか一項に記載のナノ粒子組成物。 46. The nanoparticle composition according to any one of claims 39 to 45, wherein a weight / wt ratio of the lipid component to the therapeutic and / or prophylactic agent is 10 : 1 to 60 : 1. 前記治療薬および／または予防薬に対する前記脂質成分のｗｔ／ｗｔ比が、２０：１である、請求項３９〜４６のいずれか一項に記載のナノ粒子組成物。 47. The nanoparticle composition according to any one of claims 39 to 46 , wherein the weight / wt ratio of the lipid component to the therapeutic and / or prophylactic agent is 20 : 1. 前記Ｎ：Ｐ比が、２：１〜３０：１である、請求項３９〜４７のいずれか一項に記載のナノ粒子組成物。 48. The nanoparticle composition according to any one of claims 39 to 47 , wherein the N: P ratio is 2 : 1 to 30 : 1. 請求項３９〜４８のいずれか一項に記載のナノ粒子組成物と、薬学的に許容できる担体とを含む医薬組成物。 49. A pharmaceutical composition comprising the nanoparticle composition according to any one of claims 39 to 48 and a pharmaceutically acceptable carrier. 請求項１〜２５のいずれか一項に記載の化合物を合成する方法であって、  A method for synthesizing a compound according to any one of claims 1 to 25, comprising:
前記化合物を提供するための適切な条件下で、ヘプタデカン−９−イル８−（（２−ヒドロキシエチル）アミノ）オクタノエートをノニル−８−ブロモオクタノエートと反応させることを含む、方法。  Reacting heptadecan-9-yl 8-((2-hydroxyethyl) amino) octanoate with nonyl-8-bromooctanoate under conditions suitable to provide said compound. 哺乳動物細胞への治療薬および／または予防薬の送達に使用するための請求項３９〜４８のいずれか一項に記載のナノ粒子組成物であって、該組成物は、対象に投与され、前記投与により、前記細胞がナノ粒子組成物と接触させられ、それによって、前記治療薬および／または予防薬が、前記細胞に送達される、ナノ粒子組成物。 A nanoparticle composition according to any one of claims 39 to 48 for use in the delivery of therapeutic and / or prophylactic agents into mammalian cells, the composition is administered to the subject, the administration, the cells are allowed to contact with the nanoparticle compositions, whereby the therapeutic and / or prophylactic agent is delivered into the cell, the nanoparticle composition. 哺乳動物細胞内における目的のポリペプチドの生成に使用するための請求項３９〜４８のいずれか一項に記載のナノ粒子組成物であって、該組成物は、前記細胞と接触させられ、前記治療薬および／または予防薬がｍＲＮＡであり、前記ｍＲＮＡが、前記目的のポリペプチドをコードしており、それによって、前記ｍＲＮＡが、前記細胞内で翻訳されて、前記目的のポリペプチドを生成することが可能である、ナノ粒子組成物。 49. A nanoparticle composition according to any one of claims 39 to 48 for use in the production of a polypeptide of interest in mammalian cells, wherein the composition is contacted with the cells , a therapeutic and / or prophylactic agent is mRNA, the mRNA is, encodes a polypeptide of the object, whereby, the mRNA is being translated in the cell to produce the polypeptide of the object A nanoparticle composition that is possible.
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